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Mounting scientific evidence continues to dash once-high hopes that lithium can help people with amyotrophic lateral sclerosis. On April 6, a multicenter team of researchers reported online in the journal Lancet that their double-blind, placebo-controlled trial came up negative. Lead authors were Swati Aggarwal of the Massachusetts General Hospital in Boston and Lorne Dinman of the Sunnybrook Health Sciences Center in Toronto, Canada.

The trial was started in the wake of news that Italian researchers found impressive effects from lithium in mice and in a small human cohort (see ARF related news story on Fornai et al., 2008). Aggarwal and colleagues planned their study with a built-in interim analysis once 84 subjects had enrolled. At that time, in September of 2009, a National Institute of Neurological Disorders and Stroke panel decided that more subjects, or more time, would not yield the spectacular results the study was designed to discover. ARF first covered this development in our January round-up of clinical trials news in ALS (see ARF related news story).

The trial organizers used a design that is unusual in ALS trials. Because people with ALS could get lithium off-label (it is used to treat mania), and thus would be wary about landing in the placebo group, the scientists promised that placebo participants would be switched to lithium after their condition progressed by a certain amount. This design, with built-in interim analyses and crossover from placebo, is attractive for ALS trials, wrote Michael Swash of the Royal London Hospital in an accompanying commentary, because it allows researchers to obtain results quickly.